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Brand Name :
Cerdelga
Synonyms :
eliglustat
Class :
Glucosylceramide Synthase Inhibitor; Enzyme Inhibitor
Brand Name :
Cerdelga
Synonyms :
eliglustat
Class :
Glucosylceramide Synthase Inhibitor; Enzyme Inhibitor
Dosage Forms & StrengthsÂ
CapsuleÂ
84mgÂ
Based on the patient's CYP2D6 metabolizer status, adjust the dose
CYP2D6 Extensive Metabolizer or Intermediate Metabolizer: Administer 84mg orally twice daily.
CYP2D6 Poor Metabolizer: Administer 84mg orally every day.
Safety and efficacy not establishedÂ
There needed to be more respondents aged 65 to establish if they responded differently than younger people.Â
It may enhance the effect when combined with grapefruit by CYP3A4 metabolism
may enhance the concentration of serum when combined with eliglustat
may enhance the concentration of serum when combined with eliglustat
may enhance the concentration of serum when combined with eliglustat
may enhance the concentration of serum when combined with eliglustat
may enhance the concentration of serum when combined with eliglustat
eliglustat: they may enhance the serum concentration of CYP3A Inhibitors
eliglustat: they may enhance the serum concentration of CYP3A Inhibitors
eliglustat: they may enhance the serum concentration of CYP3A Inhibitors
eliglustat: they may enhance the serum concentration of CYP3A Inhibitors
eliglustat: they may enhance the serum concentration of CYP3A Inhibitors
eliglustat: they may enhance the serum concentration of CYP2D6 Inhibitors
eliglustat: they may enhance the serum concentration of CYP2D6 Inhibitors
eliglustat: they may enhance the serum concentration of CYP2D6 Inhibitors
eliglustat: they may enhance the serum concentration of CYP2D6 Inhibitors
eliglustat: they may enhance the serum concentration of CYP2D6 Inhibitors
hepatic enzyme metabolism i.e., CYP2D6 metabolism is affected when lidocaine used combinely with eliglustat and raising its levels
when both drugs are combined, there may be an increased effect of flibanserin by affecting hepatic or intestinal enzyme cyp3a4 metabolism  
may enhance the serum concentration of CYP3A4 inhibitors
may enhance the serum concentration of CYP3A4 inhibitors
may enhance the serum concentrations of CYP2D6 inhibitors
may enhance the serum concentration of CYP3A4 Inhibitors
by affecting intestinal metabolism, the effect of eliglustat may be increased
It may enhance QTc interval when combined with lithium
nafcillin will decrease the effect of action of eliglustat by affecting enzyme CYP3A4 metabolism.
increase serum level of daunorubicin by P-glycoprotein efflux transporter
QTc interval is increased both by lenvatinib and eliglustat
lapatinib increases the effect of eliglustat by altering the intestinal or hepatic CYP3A4 enzyme metabolism
CYP3A strong enhancers of the small intestine may reduce the bioavailability of eliglustat 
when used together, entrectinib and eliglustat both increase the QTc interval
when used together, encorafenib and eliglustat both increase the QTc interval
increase serum level of idarubicin by P-glycoprotein efflux transporter
may increase the QTc interval when combined
may increase the level of effectiveness through P-glycoprotein MDR1 efflux transporter
It may enhance QTc interval when combined with pentamidine
CYP3A4 Inducers may decrease the serum concentration of eliglustat
CYP3A4 Inducers may decrease the serum concentration of eliglustat
CYP3A4 Inducers may decrease the serum concentration of eliglustat
CYP3A4 Inducers may decrease the serum concentration of eliglustat
CYP3A4 Inducers may decrease the serum concentration of eliglustat
eliglustat: they may diminish the serum concentration of CYP3A4 Inducers
It may enhance the levels when combined with tamsulosin by affecting CYP2D6 metabolism
eliglustat: it may decrease the metabolism of sulfamethoxazole
when both drugs are combined, there may be a decreased metabolism of eliglustat   
the effect of eliglustat is decreased by lorlatinib, by altering intestinal or hepatic CYP3A4 enzyme metabolism
may enhance serum levels of docetaxel by inducing MDR1/PG-P efflux transporter
Actions and Spectrum:Â
The spectrum of activity of eliglustat is specific to Gaucher disease type 1, a lysosomal storage disorder caused by mutations in the GBA gene.Â
Frequency definedÂ
>10%Â
Headache (13-40%)Â
Nausea (10-12%)Â
Back pain (12%)Â
Extremity pain (11%)Â
Arthralgia (45%)Â
Fatigue (14%)Â
Diarrhea (12%)Â
1-10%Â
Migraine (10%)Â
Oropharyngeal pain (10%)Â
Asthenia (8%)Â
Dyspepsia (7%)Â
Constipation (5%)Â
Upper abdominal pain (10%)Â
Flatulence (10%)Â
Dizziness (8%)Â
Cough (7%)Â
GERD (7%)Â
Black box warning:Â
NoneÂ
Contraindications/caution:Â
Contraindications:Â
Caution:Â
Pregnancy consideration: Uncontrolled type 1 Gaucher disease is linked to an increased chance of spontaneous miscarriage; it can also cause unfavorable pregnancy outcomes such as maternal hepatosplenomegaly and thrombocytopenia.Â
Lactation: Excretion of the drug in human breast milk is unknownÂ
Pregnancy category:Â
Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.Â
Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.Â
Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.   Â
Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.   Â
Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.   Â
Category N: There is no data available for the drug under this categoryÂ
Pharmacology:Â
eliglustat is classified as a substrate-reduction therapy. Its mechanism of action involves inhibiting the enzyme glucosylceramide synthase, which is responsible for the production of glucosylceramide. This lipid accumulates in the cells of individuals with Gaucher disease due to deficient activity of the enzyme glucocerebrosidase. By inhibiting glucosylceramide synthase, eliglustat reduces the production of glucosylceramide and subsequently decreases the accumulation of this lipid in affected cells, primarily macrophages in the spleen, liver, and bone marrow.Â
Pharmacodynamics:Â Â
The pharmacodynamics of eliglustat revolve around its ability to inhibit glucosylceramide synthase, reducing glucosylceramide accumulation in cells. This reduction improves various clinical manifestations of Gaucher disease type 1.Â
Pharmacokinetics:Â
AbsorptionÂ
The bioavailability of eliglustat is very low (<5%) due to significant first-pass metabolism. Peak plasma concentration occurs approximately 1.5 to 2 hours after dosing.Â
CYP2D6 Extensive Metabolizers (EM):Â
The Peak plasma concentration is 12.1 to 25 ng/mL.Â
AUC: 76.3 to 143 hr•ng/mL.Â
CYP2D6 Intermediate Metabolizers (IM):Â
The Peak plasma concentration is 44.6 ng/mL.Â
AUC: 306 hr•ng/mL.Â
CYP2D6 Poor Metabolizers (PM):Â
Peak plasma time: 3 hours for twice-daily dosing.Â
Peak plasma concentration: 113-137 ng/mL for twice-daily dosing, 75 ng/mL predicted for once-daily dosing.Â
AUC: 922-1057 hr•ng/mL for twice-daily dosing, 956 hr•ng/mL predicted for once-daily dosing.Â
DistributionÂ
Approximately 76-83% of eliglustat is protein-bound.Â
It is mainly distributed in plasma and not within red blood cells.Â
The volume of distribution (Vd) is high at 835 L.Â
MetabolismÂ
eliglustat is extensively metabolized in the liver.Â
The primary metabolic pathways are via CYP2D6 and, to a lesser extent CYP3A4.Â
No active metabolites have been identified.Â
Elimination and ExcretionÂ
The half-life of eliglustat is around 6.5 hours for extensive metabolizers (EM) and 8.9 hours for poor metabolizers (PM).Â
The total body clearance is high at 88 L/hr.Â
Elimination occurs primarily through the feces (51.4%) and urine (41.8%), primarily as metabolites.Â
Administration:Â
Oral administrationÂ
Do not open, crush, or dissolve capsules; swallow them whole. Â
Take it with or without a meal.Â
Avoid consuming grapefruit or grapefruit juice.Â
Patient information leafletÂ
Generic Name: eliglustatÂ
Why do we use eliglustat?Â
eliglustat is a medication used to treat Gaucher disease type 1, a rare genetic disorder. Â
Gaucher disease: It is characterized by the deficiency of an enzyme called glucocerebrosidase. This leads to the accumulation of a lipid called glucosylceramide, primarily within macrophages in various organs, including the spleen, liver, and bone marrow. eliglustat is specifically indicated for treating Gaucher disease type 1 in adult and pediatric patients.Â
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